Sat.Jun 17, 2023 - Fri.Jun 23, 2023

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First Duchenne gene therapy approved by FDA for young children

BioPharma Drive: Drug Pricing

The conditional clearance for Sarepta’s Elevidys in 4- and 5-year-olds is a milestone for research into the deadly disease, and raises the stakes of an ongoing trial that could prove how well it works.

Therapies 319
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AHA News: Beat the Heat With Hydrating Foods This Summer

Drugs.com

TUESDAY, June 20, 2023 (American Heart Association News) -- Staying hydrated can help you live a longer, healthier life. But drinking multiple glasses of water throughout the day isn't for everyone. Health experts say that's OK. While fluids are.

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Clean, sustainable fuels made 'from thin air' and plastic waste

Science Daily: Pharmacology News

Researchers have demonstrated how carbon dioxide can be captured from industrial processes -- or even directly from the air -- and transformed into clean, sustainable fuels using just the energy from the Sun.

Research 300
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Novel Oncology Target Claudin 6 Makes its Case in Solid Tumors

Fierce BioTech

Harnessing the Power Within | In the ongoing search for new cancer treatments, companies including Context Therapeutics are developing potential therapies focused on the emerging novel oncology target Claudin 6.

Therapies 111
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Bridging Innovation & Patient Care: The Growing Role of AI

Speaker: Simran Kaur, Co-founder & CEO at Tattva.Health

AI is transforming clinical trials—accelerating drug discovery, optimizing patient recruitment, and improving data analysis. But its impact goes far beyond research. As AI-driven innovation reshapes the clinical trial process, it’s also influencing broader healthcare trends, from personalized medicine to patient outcomes. Join this new webinar featuring Simran Kaur for an insightful discussion on what all of this means for the future of healthcare!

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UniQure stock tumbles on ‘confusing’ results for Huntington’s gene therapy

BioPharma Drive: Drug Pricing

While treated patients appear to be doing better than history suggests they would, mixed findings involving a key surrogate marker perplexed Wall Street analysts and sent shares falling 38%.

Therapies 294

More Trending

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Do hummingbirds drink alcohol? More often than you think

Science Daily: Pharmacology News

Animals that eat fruit or sip nectar often ingest alcohol because naturally occurring yeasts turning sugar into ethanol. But how do animals feel about that? A new study details an experiment to determine whether hummingbirds are turned off by alcohol in sugar water. At 1% by volume, no. At 2% by volume, they consume much less. The implication is that hummingbirds have adjusted to small amounts of alcohol likely present in flowers and backyard feeders.

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The genetic modifier approach: identifying the right target for rare diseases

Drug Target Review

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging. Enabling the systematic discovery of these largely uncharted targets can be a valuable opportunity for development of novel therapies.

Disease 111
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Sarepta prices Duchenne gene therapy at $3.2M

BioPharma Drive: Drug Pricing

The cost makes Sarepta’s treatment, called Elevidys, among the most expensive medicines in the world. But company executives said they don’t expect significant insurer pushback.

Therapies 287
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Legalizing Marijuana Tied to More Binge Drinking in Folks Over 30

Drugs.com

WEDNESDAY, June 21, 2023 -- A growing number of states are legalizing marijuana for recreational use, and it may be leading to an unexpected side effect among millennials and Gen Xers: binge drinking. Binge drinking is defined as five or more drinks.

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From Diagnosis to Delivery: How AI is Revolutionizing the Patient Experience

Speaker: Simran Kaur, Founder & CEO at Tattva Health Inc.

The healthcare landscape is being revolutionized by AI and cutting-edge digital technologies, reshaping how patients receive care and interact with providers. In this webinar led by Simran Kaur, we will explore how AI-driven solutions are enhancing patient communication, improving care quality, and empowering preventive and predictive medicine. You'll also learn how AI is streamlining healthcare processes, helping providers offer more efficient, personalized care and enabling faster, data-driven

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Wildfire smoke downwind affects health, wealth, mortality

Science Daily: Pharmacology News

Smoke particulates from wildfires could cause between 4,000 and 9,000 premature deaths and cost between $36 to $82 billion per year in the United States, according to new research.

Research 242
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2?Aroyl quinazolinone: Synthesis and in vitro anti?parasitic activity

Chemical Biology and Drug Design

Anti-parasitic activity of 2-aroyl quinazolinones. Abstract Trypanosomes and Leishmania are parasitic protozoans that affect millions of people globally. Herein we report the synthesis of 2-aroyl quinazolinones and their antiprotozoal efficacy against Trypanosoma brucei , Trypanosoma brucei rhodesiense , Trypanosoma cruzi , and Leishmania infantum. These compounds were counter-screened against a human cell line for cytotoxicity.

Research 100
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Flagship’s latest startup aims to take the guesswork out of small molecule drugs

BioPharma Drive: Drug Pricing

Empress Therapeutics claims its technology allows it to identify chemical drug candidates faster and more reliably. It has $50 million from Flagship to prove it can.

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FDA Orders Stores to Stop Selling Elf Bar Disposable Vapes

Drugs.com

FRIDAY, June 23, 2023 -- The U.S. Food and Drug Administration said Thursday it has sent warning letters to 189 retailers, telling them to stop selling unauthorized tobacco products such as Elf Bar and Esco Bars. Both brands are disposable.

FDA 297
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How Machine Learning Drives Clinical Trial Efficiency

Clinical trial data management is increasingly challenging as studies grow in complexity. Quickly accessing and analyzing study data is vital for assessing trial progress and patient safety. In this paper, we explore real-time data access and analysis for proactive study management. We investigate using adverse event (AE) data to monitor safety and discuss a clinical analytics platform that supports collaboration and data review workflows.

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New study reveals global reservoirs are becoming emptier

Science Daily: Pharmacology News

Over the past two decades, global reservoirs have become increasingly empty despite an overall increase in total storage capacity due to the construction of new reservoirs. Researchers used a new approach with satellite data to estimate the storage variations of 7,245 global reservoirs from 1999 to 2018.

Research 230
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Radiation in Combination with Immune Checkpoint Blockade and DNA Damage Response Inhibitors in Mice: Dosage Optimisation in MC38 Syngeneic Tumours via Modelling and Simulation [Inflammation, Immunopharmacology, and Asthma]

ASPET

Clinical trials assessing the impact of Radiotherapy (RT) in combination with inhibitors of the DNA Damage Response Pathway (DDRi) and/or immune checkpoint blockade are currently ongoing. However, current methods for optimising dosage and schedule are limited. A mathematical model was developed to capture the impacts of RT in combination with DDRi and/or anti PD-L1 (Immune Checkpoint Inhibitor, ICI) on tumour immune interactions.

DNA 100
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Cell and gene therapy manufacturing: the next generation of startups

BioPharma Drive: Drug Pricing

Joining larger CDMOs like Lonza and Catalent, a new group of companies aims to capitalize on persistent bottlenecks in producing complex genetic treatments.

Therapies 272
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Cirrhosis of the Liver: Causes, Symptoms & Treatment

Drugs.com

TUESDAY, June 20, 2023 -- Cirrhosis of the liver poses a significant health challenge worldwide. With no known cure, it requires comprehensive management to slow liver damage, alleviate symptoms and prevent potential complications. Cirrhosis is.

Treatment 295
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Deliver Fast, Flexible Clinical Trial Insights with Spotfire

Clinical research has entered a new era, one that requires real-time analytics and visualization to allow trial leaders to work collaboratively and to develop, at the click of a mouse, deep insights that enable proactive study management. Learn how Revvity Signals helps drug developers deliver clinical trial data insights in real-time using a fast and flexible data and analytics platform to empower data-driven decision-making.

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'We're all Asgardians': New clues about the origin of complex life

Science Daily: Pharmacology News

According to a new study, eukaryotes -- complex life forms with nuclei in their cells, including all the world's plants, animals, insects and fungi -- trace their roots to a common Asgard archaean ancestor. That means eukaryotes are, in the parlance of evolutionary biologists, a 'well-nested clade' within Asgard archaea, similar to how birds are one of several groups within a larger group called dinosaurs, sharing a common ancestor.

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Hsa?miR?503?5p regulates CTDSPL to accelerate cisplatin resistance and angiogenesis of lung adenocarcinoma cells

Chemical Biology and Drug Design

High expression of hsa-miR-503-5p and decreased expression of CTDSPL in LUAD contribute to cisplatin resistance and angiogenesis. Knockdown of hsa-miR-503-5p resensitizes cells to cisplatin, inhibits angiogenesis, and promotes apoptosis. Hsa-miR-503-5p negatively regulates CTDSPL, suggesting its potential as a target to overcome cisplatin resistance in LUAD.

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Patient death spurs FDA to pause test of Arcellx’s Gilead-partnered cell therapy

BioPharma Drive: Drug Pricing

The company said limitations on "bridging treatment" used to hold cancer in check could be to blame, and is working with the FDA to change the study's protocol.

FDA 263
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Is Child's Tummy Pain a Serious Concern? Poll Finds Many Parents Unsure

Drugs.com

MONDAY, June 19, 2023 -- Kids get a lot of tummy aches. How a parent responds to it can vary, just as the causes can, according to the University of Michigan Health's C.S. Mott Children’s Hospital National Poll on Children’s Health, which looked.

Hospitals 295
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Clinical Data Like You´ve Never Seen It Before: Why Spotfire Is the Leading Tool for Clinical Analytics

Clinical development organizations face a wide array of challenges when it comes to data, many of which can impact the operational effectiveness of their clinical trials. In this whitepaper, experts from Revvity Signals explore how solutions like TIBCO® Spotfire® enable better, more streamlined studies. The whitepaper also features a success story from Ambrx, a leading biopharmaceutical company, detailing how it has leveraged Spotfire to tackle data quality and collaboration challenges in clinic

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DNA can fold into complex shapes to execute new functions

Science Daily: Pharmacology News

DNA can mimic protein functions by folding into elaborate, three-dimensional structures, according to a new study.

DNA 222
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International Women in Engineering Day

NIOSH Science Blog: Drugs

June 23, 2023 is International Women in Engineering Day. In the United States, women make up 14% of the engineering workforce (15% internationally).[1] In 2020, 24% of bachelor’s degrees in engineering were earned by women, and women of color earned 10% of the total engineering degrees.[1] The National Institute for Occupational Safety and Health (NIOSH) understands the importance of diversity throughout the STEM fields and in occupational safety and health.

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Top FDA official overruled review team in approval of Sarepta’s Duchenne gene therapy

BioPharma Drive: Drug Pricing

In a memo made public Friday, Peter Marks, head of the agency office that oversees gene therapies, wrote that he disagreed with other reviewers and saw “compelling” evidence to clear Elevidys.

Therapies 256
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FDA Approves First Gene Therapy, Elevidys, to Treat Duchenne Muscular Dystrophy

Drugs.com

FRIDAY, June 23, 2023 -- The U.S. Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD).

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Regular napping linked to larger brain volume

Science Daily: Pharmacology News

A study analyzed data from people aged 40 to 69 and found a causal link between habitual napping and larger total brain volume -- a marker of good brain health linked to a lower risk of dementia and other diseases.

Disease 217